Work

The RESTORE Registry: Real-World Assessments of Interventions and Long-Term Outcomes in Patients with Spinal Muscular Atrophy (SMA) (P15-5.002)

Year: 2022

Type: article

Study design: Observational Study

Source: Neurology

DOI: 10.1212/wnl.98.18_supplement.1033

Authors: Richard Finkel, John Day, Darryl De Vivo, Eugenio Mercuri, Francesco Muntoni, Perry Shieh, Eduardo Tizzano, Isabelle Desguerre, Kayoko Saito, Melissa Menier, Nicole LaMarca, Fred Anderson, Omar Dabbous, Laurent Servais

Institutions: St. Jude Children's Research Hospital, Stanford Medicine, Stanford University, Columbia University Irving Medical Center, Pontifícia Universidade Católica de São Paulo, Great Ormond Street Hospital, NIHR Great Ormond Street Hospital Biomedical Research Centre, University College London, University of California, Los Angeles, Vall d'Hebron Hospital Universitari, Hôpital Necker-Enfants Malades, Tokyo Women's Medical University, Novartis Gene Therapies, Inc. (United States), University of Massachusetts Chan Medical School, University of Oxford

Language: English

Topic: Neurogenetic and Muscular Disorders Research

Subfield: Genetics

Field: Medicine

Domain: Health Sciences

SDG: Good health and well-being

Open Access status: closed

OpenAlex ID: W4389376919

FWCI:

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Related to: 10